Amylyx to Present Updates on its AMX0114 and RELYVRIO® Programs in ALS at the 2024 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference

CAMBRIDGE, Mass. — Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) today announced that two abstracts detailing next steps in the development of the Company’s investigational antisense oligonucleotide (ASO), AMX0114, for the potential treatment of amyotrophic lateral sclerosis (ALS) and findings from a pharmacokinetic and pharmacodynamic study of AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO]) in people with ALS will be presented at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference on March 3-6, 2024 in Orlando, Florida. AMX0035 is marketed by Amylyx as RELYVRIO^® and is approved to treat ALS in adults in the U.S. and approved with conditions as ALBRIOZA™ for the treatment of ALS in Canada.

Details of the poster presentations are as follows:

Title: Next Steps in Development for AMX0114: An Antisense Oligonucleotide Targeting Calpain-2, a Critical Effector of Axonal Degeneration
The Company is advancing AMX0114, our internally developed ASO targeting calpain-2, a critical effector of axonal degeneration in ALS and other neurodegenerative diseases, through investigational new drug enabling studies, with the goal of entering the clinic in 2024. This poster shares details on pre-clinical efficacy studies as well as introduces the design approach for the first-in-human study, anticipated to begin later this year. The Company is studying multiple cellular pathways implicated in disease pathogenesis as we believe that it is going to take a combination approach to find a cure for ALS.
Date and Time: March 4, 2024, from 6:00PM – 8:00PM EST

Title: Findings From a Pharmacokinetic and Pharmacodynamic Study of Sodium Phenylbutyrate and Taurursodiol in Participants With Amyotrophic Lateral Sclerosis
This poster provides results from a Phase 2a, open-label, sequential period study of AMX0035 pharmacokinetics in participants with ALS and includes safety data in addition to pharmacokinetics of AMX0035 and its major metabolites. No new safety signals were identified during this study.
Date and Time: March 4, 2024, from 6:00PM – 8:00PM EST

 

About RELYVRIO^®/ ALBRIOZA™ / ALBRIOZA^® / AMX0035

RELYVRIO^®, an oral, fixed-dose combination of sodium phenylbutyrate and taurursodiol (known as ursodoxicoltaurine outside of the U.S.), is approved to treat amyotrophic lateral sclerosis (ALS) in adults in the U.S. and approved with conditions as ALBRIOZA™ for the treatment of ALS in Canada. AMX0035 is being studied for the potential treatment of other neurodegenerative diseases, and Amylyx is exploring its treatment in other populations and regions. The formulation of RELYVRIO, ALBRIOZA, and AMX0035 are identical.

 

About Amylyx Pharmaceuticals

Amylyx Pharmaceuticals, Inc. is committed to supporting and creating more moments for the neurodegenerative community through the discovery and development of innovative new treatments. Amylyx is headquartered in Cambridge, Massachusetts and has operations in Canada, EMEA, and Japan.